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Taking trials to patients in rare disease clinical trials

Helen Springford

Finding and retaining patients for a clinical trial is a problem all researchers will be aware of. Studies that put the patient’s welfare first are far more likely to succeed in that respect, as Helen Springford of Illingworth Research Group explains.

The use of off-site or
‘mobile’ research nursing within clinical trials is certainly not a new
concept. That said, increasingly frequent use within clinical research points
towards a more patient-centric general trend across clinical trials.

Taking the trial to
the patient, with mobile research nurses able to perform many of the requisite
tasks in the home or the patient’s school or workplace, can drastically reduce
the levels of stress associated with travelling to the hospital for visits and
can, as a result, improve the quantity of patients a study attracts.

Conducting trials visits at patients’ homes/schools/workplaces

“We see mobile
research nurses take blood samples for pharmacokinetic sampling (measuring the
movement of drugs throughout the body), administering intravenous drugs or even
simple tasks like monitoring a patient taking an oral study medication.”

“These don’t need to be
done in a clinic and, with rare diseases specifically – where your patients are,
by the very nature of what they have, likely to be few and far between – finding
research nurses near them makes the decision of whether or not to take part in
a study a far simpler one.”

Ensuring a study does
all it can to lessen the impact on a patient’s and their family’s life can
vastly improve both patient recruitment and retention in the long-term.

An Icelandic family needed to travel to Canada
for trials; until trials mobilised

“A family from
Reykjavik in Iceland had two young boys with Duchenne Muscular Dystrophy (DMD –
a rare genetic disorder),” Helen explains.

“They found out about
a trial that could benefit them, the only problem being the site was based
within a hospital in Calgary, Canada.”

With frequent visits
over eight weeks, participation would have meant either staying in Canada or
travelling back and forth – neither being an option for the family. Conducting
a portion of the visits at the family home in Iceland through off-site research
nurses meant the boys could take part in the study.

Another of Helen’s
stories really brings home how important flexibility is becoming to patients
when making a tough decision on whether or not to participate in a trial.

Knowing it would be her last Christmas, this
patient wanted to be at home

“One terminally ill
patient told me that she was desperate to perform her yearly ritual of making the
Christmas puddings for her children on what she knew would be her last
Christmas with them. Had it not been possible for the visits to take place at
her home, taking part would have left her too exhausted to be a part of the
study and achieve the things she wanted to with her remaining time.”

The study of rare
diseases poses many challenges, yet engaging patients through clinical trials that
are designed to lessen the negative impact on their lives seems to be a way to
alleviate one of the more pressing ones – finding patients and retaining them.